One of the world's top ex­perts in coro­nary heart dis­ease is spear­head­ing a new gene edit­ing up­start out to trans­form the field

As head of the Cen­ter for Hu­man Ge­net­ic Re­search at Mass­a­chu­setts Gen­er­al Hos­pi­tal and the Broad’s Car­dio­vas­cu­lar Dis­ease Ini­tia­tive, Sekar Kathire­san has oc­cu­pied a sin­gu­lar po­si­tion as one of the world’s lead­ing ex­perts on the con­nec­tion be­tween ge­net­ics and coro­nary heart dis­ease. He’s tracked how peo­ple dealt a bad ge­net­ic hand — and the el­e­vat­ed risks that come with it — can lim­it in­her­ent dan­gers by lifestyle changes, and pon­dered over the ef­fects of dai­ly drugs used to treat mass pa­tient groups. And he’s reached a sim­ple con­clu­sion:

None of it is re­al­ly work­ing. 

In par­tic­u­lar, none of that is any­where near as use­ful as the ge­net­ic mu­ta­tions that he’s seen that con­fer a low­er risk of dy­ing and car­dio events. Par­tic­u­lar­ly the in­di­vid­u­als who car­ry mu­ta­tions “which break ei­ther of two genes — APOC3 or ANGPTL3 — rapid­ly clear triglyc­eride-rich lipopro­teins from the cir­cu­la­tion” and pro­tect them from heart at­tack.

Now, he’s mak­ing a pro­fes­sion­al leap to see if he and a squad of in­ves­ti­ga­tors at a new­ly launched biotech can dra­mat­i­cal­ly change the im­per­fect sta­tus quo through gene edit­ing.

“Imag­ine,” he tells me, “an in­jec­tion ad­min­is­tered once in life that safe­ly con­fers last­ing pro­tec­tion.”

No more pills. No fleet­ing com­mit­ments to healthy lifestyles that can’t stretch past Jan­u­ary. But a wide­spread and durable shar­ing of the same health ben­e­fits he’s seen in the very, very few. That’s the dream.

To­day, Kathire­san is step­ping down from his lofty aca­d­e­m­ic roles and mak­ing his de­but as CEO of Verve Ther­a­peu­tics, which is tak­ing its place in the hotbed of gene ther­a­py work around Cam­bridge, MA. The small team of 10 — soon to dou­ble in size — may not come close to ri­val­ing the big biotechs that oc­cu­py the streets in and around Har­vard and MIT. But rel­a­tive­ly few can claim the same kinds of con­nec­tions in the realms of drug sci­ence.

Ki­ran Musunuru

An­oth­er car­dio ge­net­ics ex­pert, Penn’s Ki­ran Musunuru and Har­vard pro­fes­sor J Kei­th Joung, who co-found­ed gene ther­a­py pi­o­neer Ed­i­tas, are on board as sci­en­tif­ic co-founders. There’s an al­liance with Beam Ther­a­peu­tics, the up­start next-gen gene edit­ing out­fit found­ed by Feng Zhang, one of 3 sci­en­tists wide­ly cred­it­ed with ush­er­ing in the CRISPR rev­o­lu­tion that has armed re­searchers around the world with ef­fec­tive tools to ac­com­plish their work. And Ver­i­ly will help work on the nanopar­ti­cles they plan to use for de­liv­ery.

Burt Adel­man, the for­mer EVP of R&D at Bio­gen, will chair the board, which in­cludes the Broad’s chief da­ta of­fi­cer, An­tho­ny Philip­pakis.

Beam will pro­vide some of the tech, and has an op­tion to step in on fu­ture com­mer­cial­iza­tion. Verve has al­so nailed down CRISPR patents, in­clud­ing Cas9 and Cas12a (Cpf1), from the Broad In­sti­tute and Har­vard Uni­ver­si­ty.

And they have $58.5 mil­lion in cash to do their work from GV (you prob­a­bly still think of them as Google Ven­tures), which is step­ping in with ARCH Ven­ture Part­ners, F-Prime Cap­i­tal, and Bio­mat­ics Cap­i­tal to form the orig­i­nal syn­di­cate.

To be suc­cess­ful, the Verve team un­der Kathire­san will not just have to demon­strate their ap­proach can safe­ly work, but al­so that it ul­ti­mate­ly can be done on a mass ba­sis in eco­nom­ic terms. That’s a tall or­der, but they do have some ad­van­tages, per­haps most no­tice­ably the ad­vances the ground­break­ers have made at the FDA, says the sci­en­tist.

“Gene edit­ing has the po­ten­tial to com­plete­ly trans­form the treat­ment par­a­digm for the dis­ease,” says Musunuru. “Pre­clin­i­cal stud­ies con­duct­ed in the field, in­clud­ing work done in my lab, have shown the promise of gene edit­ing to safe­ly re­duce cho­les­terol and oth­er coro­nary artery dis­ease risk fac­tors.”

So far, gene edit­ing in the le­git­i­mate R&D world has been cen­tered on the painstak­ing ad­vances of a hand­ful of pro­grams aimed at rare dis­eases. And Verve will start with its own rare ail­ments, tar­get­ing pa­tient pop­u­la­tions with the high­est un­met med­ical need. But Kathire­san isn’t drop­ping his pres­ti­gious aca­d­e­m­ic roles to search for mar­gin­al gains. He wants to tack­le the whole threat on a world­wide ba­sis.

That, of­fi­cial­ly, starts at Verve to­day.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of face plants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Maureen Hillenmeyer, Hexagon Bio CEO

Hexa­gon Bio rais­es $61M to con­tin­ue ef­forts to turn fun­gi in­to drugs

A year after raising a $47 million launch round, the fungi-loving drug hunters at Hexagon Bio have more than doubled their coffers.

Hexagon announced today that it raised another $61 million for its efforts to design cancer and infectious disease drugs based on insights mined from the DNA in millions of species of fungi. The new financing brings Hexagon’s committed funding to over $108 million.